Does biomarker use in oncology improve clinical trial failure risk? A large-scale analysis

Jayson L. Parker, Sebnem S. Kuzulugil, Kirill Pereverzev, Stephen Mac, Gilberto Lopes, Zain Shah, Ashini Weerasinghe, Daniel Rubinger, Adam Falconi, Ayse Bener, Bora Caglayan, Rohan Tangri, Nicholas Mitsakakis

Research output: Contribution to journalArticlepeer-review

1 Scopus citations


Purpose: To date there has not been an extensive analysis of the outcomes of biomarker use in oncology. Methods: Data were pooled across four indications in oncology drawing upon trial outcomes from breast cancer, non-small cell lung cancer (NSCLC), melanoma and colorectal cancer from 1998 to 2017. We compared the likelihood drugs would progress through the stages of clinical trial testing to approval based on biomarker status. This was done with multi-state Markov models, tools that describe the stochastic process in which subjects move among a finite number of states. Results: Over 10000 trials were screened, which yielded 745 drugs. The inclusion of biomarker status as a covariate significantly improved the fit of the Markov model in describing the drug trajectories through clinical trial testing stages. Hazard ratios based on the Markov models revealed the likelihood of drug approval with biomarkers having nearly a fivefold increase for all indications combined. A 12, 8 and 7-fold hazard ratio was observed for breast cancer, melanoma and NSCLC, respectively. Markov models with exploratory biomarkers outperformed Markov models with no biomarkers. Conclusion: This is the first systematic statistical evidence that biomarkers clearly increase clinical trial success rates in three different indications in oncology. Also, exploratory biomarkers, long before they are properly validated, appear to improve success rates in oncology. This supports early and aggressive adoption of biomarkers in oncology clinical trials.

Original languageEnglish (US)
Pages (from-to)1955-1963
Number of pages9
JournalCancer Medicine
Issue number6
StatePublished - Mar 2021


  • biomarkers
  • breast cancer
  • cancer
  • clinical trial
  • drug development
  • lung cancer
  • melanoma
  • oncology
  • risk

ASJC Scopus subject areas

  • Oncology
  • Radiology Nuclear Medicine and imaging
  • Cancer Research


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